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艾美赛珠单抗治疗伴有同种抗体的 3 型血管性血友病患儿的疗效。

Efficacy of emicizumab in a pediatric patient with type 3 von Willebrand disease and alloantibodies.

机构信息

Division of Hematology and Oncology, Department of Pediatrics, University of Michigan, Ann Arbor, MI.

Blood Research Institute, Blood Center of Wisconsin, Milwaukee, WI.

出版信息

Blood Adv. 2019 Sep 24;3(18):2748-2750. doi: 10.1182/bloodadvances.2019000656.

DOI:10.1182/bloodadvances.2019000656
PMID:31540901
原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC6759735/
Abstract

Type 3 von Willebrand disease with alloantibodies is a rare clinical entity with few treatment options. Emicizumab prophylaxis in such patients may result in improved hemarthrosis control, lower cost, and enhanced quality of life.

摘要

3 型血管性血友病伴同种抗体是一种罕见的临床病症,治疗选择有限。此类患者接受emicizumab 预防治疗可能会改善关节积血控制、降低成本并提高生活质量。

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Efficacy of emicizumab in a pediatric patient with type 3 von Willebrand disease and alloantibodies.艾美赛珠单抗治疗伴有同种抗体的 3 型血管性血友病患儿的疗效。
Blood Adv. 2019 Sep 24;3(18):2748-2750. doi: 10.1182/bloodadvances.2019000656.
2
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本文引用的文献

1
Safety analysis of rFVIIa with emicizumab dosing in congenital hemophilia A with inhibitors: Experience from the HAVEN clinical program.rFVIIa 与emicizumab 给药用于伴抑制物的先天性血友病 A 的安全性分析:来自 HAVEN 临床项目的经验。
J Thromb Haemost. 2019 Sep;17(9):1470-1477. doi: 10.1111/jth.14491. Epub 2019 Jun 17.
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New therapies for hemophilia.血友病的新疗法。
Blood. 2019 Jan 31;133(5):389-398. doi: 10.1182/blood-2018-08-872291. Epub 2018 Dec 17.
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Novel applications of array comparative genomic hybridization in molecular diagnostics.阵列比较基因组杂交在分子诊断中的新应用。
Expert Rev Mol Diagn. 2018 Jun;18(6):531-542. doi: 10.1080/14737159.2018.1479253. Epub 2018 May 31.
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Alloantibodies in von Willebrand Disease.血管性血友病中的同种抗体。
Semin Thromb Hemost. 2018 Sep;44(6):590-594. doi: 10.1055/s-0037-1607440. Epub 2017 Nov 17.
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Alloantibodies in von Willebrand disease.血管性血友病中的同种抗体。
Blood. 2013 Aug 1;122(5):636-40. doi: 10.1182/blood-2012-10-462085. Epub 2013 Jan 7.
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The challenging management of a child with type 3 von Willebrand disease and antibodies to von Willebrand factor.3型血管性血友病合并血管性血友病因子抗体患儿的挑战性管理
Haemophilia. 2012 May;18(3):e66-7. doi: 10.1111/j.1365-2516.2012.02799.x.
7
Critical von Willebrand factor A1 domain residues influence type VI collagen binding.关键的血管性血友病因子 A1 结构域残基影响与 VI 型胶原的结合。
J Thromb Haemost. 2012 Jul;10(7):1417-24. doi: 10.1111/j.1538-7836.2012.04746.x.
8
Clinical and molecular markers of inherited von Willebrand disease type 3: are deletions of the VWF gene associated with alloantibodies to VWF?遗传性3型血管性血友病的临床和分子标志物:VWF基因缺失是否与VWF同种抗体相关?
J Thromb Haemost. 2008 Oct;6(10):1726-8. doi: 10.1111/j.1538-7836.2008.03146.x.
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Congenital von Willebrand disease type 3: clinical manifestations, pathophysiology and molecular biology.
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Alloantibody from a patient with severe von Willebrand disease inhibits von Willebrand factor-FVIII interaction.一名患有严重血管性血友病的患者的同种抗体可抑制血管性血友病因子与凝血因子VIII的相互作用。
Ann Hematol. 1997 Sep;75(3):111-5. doi: 10.1007/s002770050323.